Trial Outcomes & Findings for A Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia (NCT NCT02804178)
NCT ID: NCT02804178
Last Updated: 2021-03-10
Results Overview
17-hydroxyprogesterone was measured predose in the morning at the beginning and end of each dose level.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
10 participants
Primary outcome timeframe
Evaluated at baseline and day 15 of each dose level. Each subject will have up to 5 dose levels.
Results posted on
2021-03-10
Participant Flow
Participant milestones
| Measure |
ATR-101
Ascending dose levels of ATR-101 beginning with 125 mg by mouth twice per day up to 1000 mg twice per day.
|
|---|---|
|
Overall Study
STARTED
|
10
|
|
Overall Study
COMPLETED
|
9
|
|
Overall Study
NOT COMPLETED
|
1
|
Reasons for withdrawal
| Measure |
ATR-101
Ascending dose levels of ATR-101 beginning with 125 mg by mouth twice per day up to 1000 mg twice per day.
|
|---|---|
|
Overall Study
Adverse Event
|
1
|
Baseline Characteristics
A Study of ATR-101 for the Treatment of Congenital Adrenal Hyperplasia
Baseline characteristics by cohort
| Measure |
ATR-101
n=10 Participants
Ascending dose levels of ATR-101 beginning with 125 mg by mouth twice per day up to 1000 mg twice per day.
|
|---|---|
|
Age, Continuous
|
30.3 years
STANDARD_DEVIATION 13.76 • n=93 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=93 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
1 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
9 Participants
n=93 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Asian
|
1 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
White
|
9 Participants
n=93 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=93 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=93 Participants
|
|
Region of Enrollment
United States
|
10 participants
n=93 Participants
|
|
17-hydroxyprogesterone
|
6591.0 ng/dL
STANDARD_DEVIATION 5371.80 • n=93 Participants
|
PRIMARY outcome
Timeframe: Evaluated at baseline and day 15 of each dose level. Each subject will have up to 5 dose levels.17-hydroxyprogesterone was measured predose in the morning at the beginning and end of each dose level.
Outcome measures
| Measure |
ATR-101
n=10 Participants
Ascending dose levels of ATR-101 beginning with 125 mg by mouth twice per day up to 1000 mg twice per day.
|
|---|---|
|
Number of Participants With Reduction of 17-hydroxyprogesterone to </= 2 Times the Upper Limit of Normal at Any Time Following 2 Weeks of Dosing With ATR-101
|
2 Participants
|
Adverse Events
ATR-101
Serious events: 1 serious events
Other events: 9 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
ATR-101
n=10 participants at risk
Ascending dose levels of ATR-101 beginning with 125 mg by mouth twice per day up to 1000 mg twice per day.
|
|---|---|
|
Gastrointestinal disorders
Enteritis
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
Other adverse events
| Measure |
ATR-101
n=10 participants at risk
Ascending dose levels of ATR-101 beginning with 125 mg by mouth twice per day up to 1000 mg twice per day.
|
|---|---|
|
Cardiac disorders
Atrioventricular block first degree
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Gastrointestinal disorders
Abdominal pain upper
|
30.0%
3/10 • Number of events 3 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Gastrointestinal disorders
Diarrhoea
|
30.0%
3/10 • Number of events 3 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Gastrointestinal disorders
Vomiting
|
20.0%
2/10 • Number of events 2 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Gastrointestinal disorders
Dyspepsia
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Gastrointestinal disorders
Gingival recession
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
General disorders
Fatigue
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
General disorders
Oedema peripheral
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Infections and infestations
Nasopharyngitis
|
40.0%
4/10 • Number of events 4 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Infections and infestations
Gastroenteritis viral
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Infections and infestations
Tooth infection
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Infections and infestations
Upper respiratory tract infection
|
10.0%
1/10 • Number of events 2 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Infections and infestations
Urinary tract infection
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Infections and infestations
Viral infection
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Injury, poisoning and procedural complications
Muscle strain
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Investigations
Weight decreased
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Metabolism and nutrition disorders
Decreased appetite
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Metabolism and nutrition disorders
Hypokalemia
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Metabolism and nutrition disorders
Hyponatremia
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Musculoskeletal and connective tissue disorders
Musculoskeletal pain
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Nervous system disorders
Headache
|
40.0%
4/10 • Number of events 5 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Nervous system disorders
Migraine
|
30.0%
3/10 • Number of events 3 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Nervous system disorders
Dizziness
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Nervous system disorders
Somnolence
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Psychiatric disorders
Depression
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Psychiatric disorders
Insomnia
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Respiratory, thoracic and mediastinal disorders
Nasal congestion
|
20.0%
2/10 • Number of events 2 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Respiratory, thoracic and mediastinal disorders
Dysphonia
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Respiratory, thoracic and mediastinal disorders
Oropharyngeal pain
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Skin and subcutaneous tissue disorders
Acne
|
20.0%
2/10 • Number of events 2 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Skin and subcutaneous tissue disorders
Pruritis generalised
|
10.0%
1/10 • Number of events 1 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
|
Skin and subcutaneous tissue disorders
Rash
|
10.0%
1/10 • Number of events 2 • Adverse events were collected from the time the patient signed informed consent until 4 weeks after the last dose of active drug.
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place